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UA127455C2 - Капсид аденоасоційованого вірусу і спосіб його застосування - Google Patents

Капсид аденоасоційованого вірусу і спосіб його застосування Download PDF

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Publication number
UA127455C2
UA127455C2 UAA202002445A UAA202002445A UA127455C2 UA 127455 C2 UA127455 C2 UA 127455C2 UA A202002445 A UAA202002445 A UA A202002445A UA A202002445 A UAA202002445 A UA A202002445A UA 127455 C2 UA127455 C2 UA 127455C2
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UA
Ukraine
Prior art keywords
aam
amino acid
capsid
acid sequence
variant
Prior art date
Application number
UAA202002445A
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English (en)
Ukrainian (uk)
Inventor
Девід Г. Керн
Девид Г. Керн
Мелісса Коттерман
Мелисса КОТТЕРМАН
Девід Шаффер
Девид Шаффер
Original Assignee
4Д Молекьюлар Терапьютикс Інк.
4Д Молекьюлар Терапьютикс Инк.
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Publication date
Application filed by 4Д Молекьюлар Терапьютикс Інк., 4Д Молекьюлар Терапьютикс Инк. filed Critical 4Д Молекьюлар Терапьютикс Інк.
Publication of UA127455C2 publication Critical patent/UA127455C2/uk

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/03—Herpetoviridae, e.g. pseudorabies virus
    • C07K14/05—Epstein-Barr virus
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/861—Adenoviral vectors
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y116/00—Oxidoreductases oxidizing metal ions (1.16)
    • C12Y116/03—Oxidoreductases oxidizing metal ions (1.16) with oxygen as acceptor (1.16.3)
    • C12Y116/03001—Ferroxidase (1.16.3.1), i.e. ceruloplasmin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01001—Phosphorylase (2.4.1.1)
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
UAA202002445A 2017-09-20 2018-09-19 Капсид аденоасоційованого вірусу і спосіб його застосування UA127455C2 (uk)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762560901P 2017-09-20 2017-09-20
PCT/US2018/051812 WO2019060454A2 (fr) 2017-09-20 2018-09-19 Virus adéno-associé à variant de capsides et leurs procédés d'utilisation

Publications (1)

Publication Number Publication Date
UA127455C2 true UA127455C2 (uk) 2023-08-30

Family

ID=65810888

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Application Number Title Priority Date Filing Date
UAA202002445A UA127455C2 (uk) 2017-09-20 2018-09-19 Капсид аденоасоційованого вірусу і спосіб його застосування

Country Status (25)

Country Link
US (3) US11118192B2 (fr)
EP (2) EP3684423B1 (fr)
JP (1) JP6825167B2 (fr)
KR (3) KR102743219B1 (fr)
CN (1) CN111836649A (fr)
AU (1) AU2018337833B2 (fr)
BR (1) BR112020005436B1 (fr)
CA (1) CA3076348A1 (fr)
CL (1) CL2020000728A1 (fr)
CO (1) CO2020004738A2 (fr)
CR (1) CR20200165A (fr)
DK (1) DK3684423T5 (fr)
ES (1) ES2948902T3 (fr)
FI (1) FI3684423T3 (fr)
IL (1) IL273261B1 (fr)
MX (1) MX2020003176A (fr)
NZ (1) NZ763018A (fr)
PE (1) PE20201264A1 (fr)
PH (1) PH12020550094A1 (fr)
PT (1) PT3684423T (fr)
RU (1) RU2770922C2 (fr)
SG (1) SG11202002276VA (fr)
UA (1) UA127455C2 (fr)
WO (1) WO2019060454A2 (fr)
ZA (1) ZA202001679B (fr)

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JP7348844B2 (ja) 2017-06-07 2023-09-21 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 内部移行酵素のための組成物および方法
KR102743219B1 (ko) 2017-09-20 2024-12-17 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 그 용도
FI3717636T3 (fi) 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
CN113164556A (zh) 2018-08-30 2021-07-23 特纳亚治疗股份有限公司 用心肌蛋白和ascl1进行的心脏细胞重编程
WO2020068990A1 (fr) 2018-09-26 2020-04-02 California Institute Of Technology Compositions de virus adéno-associé pour une thérapie génique ciblée
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
WO2020198233A1 (fr) * 2019-03-25 2020-10-01 The Regents Of The University Of California Procédés de traitement de cardiomyopathie médiée par tnni3
EP3947700A4 (fr) * 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Virus adéno-associé comprenant une capside modifiée
WO2021007515A1 (fr) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Reprogrammation de cellules cardiaques avec des microarn et d'autres facteurs
JP2022549140A (ja) * 2019-09-20 2022-11-24 ユーシーエル ビジネス リミテッド 遺伝子療法組成物および不整脈源性右心室心筋症の治療
KR20220105158A (ko) * 2019-10-23 2022-07-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
BR112022009864A2 (pt) * 2019-11-22 2022-08-02 Childrens Hospital Philadelphia Variantes do vetor viral adenoassociado
BR112022015979A2 (pt) 2020-02-13 2022-10-11 Tenaya Therapeutics Inc Vetores de terapia genética para tratamento de doenças cardíacas
US20230108316A1 (en) * 2020-04-08 2023-04-06 University Of Utah Research Foundation Methods for rehabilitating heart failure using gene therapy
WO2021222094A1 (fr) * 2020-04-27 2021-11-04 4D Molecular Therapeutics Inc. Gènes gla à codons optimisés et leurs utilisations
AU2021273447A1 (en) 2020-05-13 2022-12-08 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
US11900272B2 (en) 2020-05-13 2024-02-13 Factset Research System Inc. Method and system for mapping labels in standardized tables using machine learning
EP4149519A4 (fr) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania Compositions utiles dans le traitement de la maladie de pompe
US20230226221A1 (en) * 2020-06-30 2023-07-20 Children's National Medical Center Use of recombinant human acid sphingomyelinase to improve skeletal myofiber repair
US20230313186A1 (en) * 2020-07-24 2023-10-05 The Trustees Of The University Of Pennsylvania Trophin genome editing for treating duchenne muscular dystrophy (dmd)
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
WO2022103766A2 (fr) * 2020-11-10 2022-05-19 Avirmax, Inc. Capsides viraux modifiés et méthodes d'utilisation
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